Sangamo is developing engineered transcription factors for the regulation and modification of genes. Our proprietary technology platform is based upon the engineering of a naturally occurring class of DNA transcription factors referred to as zinc finger DNA-binding proteins, or ZFPs. The DNA recognition and binding function of ZFPs can be used to target a variety of functional domains to a gene-specific location. Sangamo scientists engineer ZFP transcription factors, or ZFP TFs, that are able to regulate genes in a targeted fashion by fusing ZFPs to certain functional domains that either turn genes on or off. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment for infectious diseases, such as HIV and a variety of monogenic diseases, such as hemophilia, Huntington's disease, lysosomal storage disroders and hemoglobinopathies such as sickle cell disease and beta-thalassemia. We believe that our ZFP technology is broadly applicable and are using it to develop novel human therapeutics and for applications in plant agriculture,cell engineering and research reagents .
ZFPs are zinc finger DNA binding proteins, a class of naturally occurring proteins recognize and bind to DNA. When these domains are fused to functional domains that normally activate or repress gene expression, we can create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. Sangamo scientists can engineer these proteins to bind to a specific DNA sequence in order to regulate the expression of a chosen target gene in any cell type. They can also engineer ZFP nucleases, or ZFNs, that can be used to facilitate therapeutic gene modification. ZFNs can be used for both disruption of a gene, such as CCR5, that facilitates a disease pathology, in this case HIV infection, or correction of a mutation in a gene that causes disease, such as the monogenic disease beta-thalassemia that is caused by a mutation in the beta-globin gene.
We believe our ZFP technology has potential application in the treatment of human diseases through the development of both ZFP TF and ZFN-based ZFP Therapeutics. In addition, with Dow AgroSciences we are applying our platform in plant agriculture and with Sigma-Aldrich Corporation as research reagents, in transgenic animal production and engineering of cell lines with enhanced characteristics for production of protein pharmaceuticals.
Internally, we are actively pursuing commercial applications of our ZFP TF and ZFN technology in human therapeutics, and to improve pharmaceutical protein production.
ZFP Therapeutics® . ZFP TFs and ZFNs have the potential to be developed as pharmaceutical products to treat a broad spectrum of diseases through the regulation of disease-related genes and the modification of disease-causing genes in patients. We have an agreeement to develop ZFP Therapeutics for hemophilia, Huntington's disease and other monogenic diseases with Shire AG. We are also developing ZFP-based medicines for our own pipeline in HIV/AIDS and monogenic diseases such as hemoglobinopathies and lysosomal storage disorders.
In June 2008, Dow AgroSciences exercised its option to become the exclusive licensee and sublicensor of Sangamo's proprietary ZFP technology for the development of products in plants and plant cell cultures following a successful research collaboration that began in October 2005.
In July 2007, we entered into an license agreement with Sigma-Aldrich Corporation that gives Sigma access to our ZFP technology and the exclusive right to develop research reagents, products and services.
Cell Engineering Applications in the Manufacturing of Protein Pharmaceuticals
ZFP TF-and ZFN-engineered cell lines can be developed to enhance production yields of protein pharmaceuticals such as monoclonal antibodies. We have research agreements with several companies including Pfizer and Genentech.
Transgenic Animal Production
We have agreements with Open Monoclonal Technologies (a private company based in California) and Hoffmann LaRoche for the use of ZFNs in transgenic animal generation.
Sangamo has licensed intellectual property directed to the design, selection and use of ZFPs and ZFP TFs for gene regulation from the Massachusetts Institute of Technology, Johnson & Johnson, The Scripps Research Institute, Harvard University and Johns Hopkins University. These licenses grant us rights to make, use and sell ZFPs, ZFNs and ZFP TFs under fifteen families of patent filings. All of these patent families have been filed in the United States and many have been filed internationally in selected countries. As of February 1, 2013 these patent filings have resulted in twenty-one issued U.S. patents. We believe these licensed patents and patent applications include all of the early and important patent filings directed to design, selection and use of ZFPs, ZFNs and ZFP TFs. As of February 1, 2013 we have one hundred and seven families of internally generated U.S. patent filings, including eighty-four U.S. and two hundred and twenty-one foreign issued patents, based on Sangamo's internal research. These patent filings are directed to improvements in the design and use of ZFPs, ZFNs and ZFP TFs and TALE proteins.
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